Accelerating Product Development for Pediatric Systemic Lupus Erythematosus (SLE)

Dates: July 30 & 31 at the FDA White Oak Campus

Background:

Systemic lupus erythematosus (SLE) is a chronic, multisystem autoimmune disease characterized by inflammation, autoantibody production, and alternating periods of disease flares and remission. Approximately 10–20% of cases have onset during childhood. Currently, only one biologic therapy is approved for pediatric SLE, leaving a substantial unmet need for additional safe and effective treatment options.

Conducting clinical trials in pediatric SLE presents several challenges, including a small patient population, limited specialized centers, and difficulties with recruitment and retention. In addition, there is a need to evaluate how existing data, often from adult SLE studies, may be appropriately extrapolated to pediatric populations to streamline drug development and reduce the burden of clinical research in pediatric patients with SLE to ensure timely availability of safe and effective therapies in this population.

Day 1 Workshop Details | July 30, 2026

Welcoming and Introduction
9:00 AM – 9:05 AM Welcome and Overview Scope of Workshop	Speaker: Lily Mulugeta, PharmD Associate Director, DPMH, CDER, FDA	Presentation(PDF)
9:05 AM – 9:15 AM Introductory Remarks	Speaker: Mary Tran Thanh Hai, MD Office Director, OND, CDER, FDA
9:15 AM – 9:30 AM Keynote Talk	Speaker: Mckenna Bowes, BS Patient Perspective
9:30 AM – 9:45 AM Regulatory Framework for Drug Development and Pediatric Extrapolation	Speaker: Lily Mulugeta, PharmD Associate Director, DPMH, CDER, FDA
9:45 AM – 10:15 AM Landscape of Pediatric SLE Drug Development: Regulatory Perspective	Speakers: Emily C. Gotschlich, MD Clinical Reviewer, DRTM, CDER, FDA Roberto De Lisa, MD, MSc Clinical Pharmacologist; Scientific Officer at European Medicines Agency (EMA)-Paediatric Medicines	Presentation (PDF) Presentation(PDF)
Session 1: Evaluation of Disease in Adult and Pediatric SLE
10:15 AM – 10:45 AM Pediatric SLE Presentation and Relationship to Adult SLE: Clinical and Pathophysiological Considerations	Speakers: Stephen Balevic, MD, PhD, MHS Associate Professor, Departments of Medicine and Pediatrics, Duke Universit Rebecca Sadun, MD, PhD Associate Professor, Departments of Medicine and Pediatrics, Duke University Stacy Ardoin, MD Professor, Pediatric and Adult Rheumatology, Nationwide Children’s Hospital, The Ohio State University College of Medicine	Presentation (PDF)
10:45 AM – 11:00 AM	Break
11:00 AM – 11:15 PM Treatment Paradigm, Landscape of Products Used, and Unmet Needs in Pediatric SLE	Speaker: Ekemini A. Ogbu MD, MSc Associate Professor of Pediatrics, University of Cincinnati, Director, Neuroinflammatory Disease Services in Rheumatology; Co-director, Cincinnati Children’s Lupus Center; Cincinnati Children’s Hospital Medical Center
11:15 AM – 12:00 PM Panel Discussion	Moderator: Suzette Peng, MD Medical Reviewer, DRTM, CDER, FDA Panelists: Tresa Ambooken, MD Medical Officer (Clinical Reviewer), FDA Rebecca Sadun, MD, PhD Associate Professor, Departments of Medicine and Pediatrics, Duke University Stacy Ardoin, MD Professor, Pediatric and Adult Rheumatology, Nationwide Children’s Hospital, The Ohio State University College of Medicine Ekemini A. Ogbu MD, MSc Associate Professor of Pediatrics, University of Cincinnati, Director, Neuroinflammatory Disease Services in Rheumatology; Co-director, Cincinnati Children’s Lupus Center; Cincinnati Children’s Hospital Medical Center Alvina Chu, MD Head of Mid-Stage Programs; Immunology Clinical Development, AbbVie Reshma Patel, MD Global Associate Medical Director, Lupus, Biogen Miah Andrade Patient/Family Representative Elizabeth SantaCruz, BS Patient/Family Representative
12:00 PM – 1:00 PM	Lunch
Session 2: Evaluation of Disease in Adult and Pediatric SLE
1:00 PM – 1:15 PM Response Similarity Between Adult and Pediatric SLE: Lessons Learned from Belimumab	Speakers: Tao Liu, PhD Team Leader, Division of Inflammation and Immune Pharmacology, Office of Clinical Pharmacology, FDA Satyajit Ghosh, PhD Mathematical Statistician, DB II, OB, CDER, FDA	Presentation(PDF)
1:15 PM – 1:30 PM Similarities and Differences of Response and Exposure-Response	Speaker: Stephen Balevic, MD, PhD, MHS Associate Professor, Departments of Medicine and Pediatrics, Duke University	Presentation (PDF)
1:30 PM – 1:45 PM Role of MIDD to Accelerate Dose Selection and Optimization	Speaker: Vikram Sinha, PhD Senior Vice President, Translational and Quantitative Medicine, Flagship Pioneering	Presentation (PDF)
1:45 PM – 2:30 PM Panel Discussion	Moderator: Lily Mulugeta, PharmD Associate Director, DPMH, CDER, FDA Panelists: Jianmeng Chen, MD, PhD Associate Director, Division of Inflammation and Immune Pharmacology Clinical Pharmacology, Office of Clinical Pharmacology, FDA James Travis, PhD Master Statistician, Office of Biostatistics, FDA Amit Golding, MD, PhD Lead Physician, DRTM, CDER, FDA Flora Musuamba Tshinanu, PhD Pharmacometrics and Pharmacovigilance Internal Expert, Belgian Federal Medicines Agency (FAMHP), European Medicines Agency (EMA) Stephen Balevic, MD, PhD, MHS Associate Professor, Departments of Medicine and Pediatrics, Duke University Vikram Sinha, PhD Senior Vice President, Translational and Quantitative Medicine, Flagship Pioneering Laura E. Schanberg, MD Professor, Pediatrics, Duke Clinical Research Institute, Duke University School of Medicine Mckenna Bowes, BS Patient Perspective
2:30 PM – 2:45 PM	Break
Sesson 3: Extrapolation of Efficacy
2:45 PM – 3:15 PM Lessons Learned from Conducting Clinical Trials in Pediatric SLE	Speaker: Hermine I. Brunner, MD, MSc, MBA Professor of Pediatrics University of Cincinnati; Director, Division of Rheumatology at Cincinnati Children’s Hospital Sule Yavuz, MD Global Clinical Program Lead, AstraZenec
3:15 PM – 3:35 PM Patient/Family Panel: Risk Tolerance and Enrollment in Clinical Trials	Moderator: Stacy Ardoin, MD Professor, Pediatric and Adult Rheumatology, Nationwide Children’s Hospital, The Ohio State University College of Medicine Panelists: Mckenna Bowes, BS Patient Representative Chloe Raymond Patient Representative Nia Cooper Patient Representative Miah Andrade Patient/Family Representative Elizabeth SantaCruz BS Patient/Family Representative
3:35 PM – 3:50 PM Extrapolation of Efficacy from Adults: Gaps in Knowledge and Remaining Uncertainti	Speaker: Rebecca Sadun, MD, PhD Associate Professor, Departments of Medicine and Pediatrics, Duke University	Presentation (PDF)
3:50 PM – 4:30 PM Panel Discussion and Q/A	Speaker: Moderators: Laura E. Schanberg, MD Professor, Pediatrics, Duke Clinical Research Institute, Duke University School of Medicine Panelists: Lily Mulugeta, PharmD Associate Director, DPMH, CDER, FDA Raj Nair, MD Division Director, DRTM, CDER, FDA Caroline Benichou-Auriche, MD EMA-Senior Clinical Expert, Icelandic Medicines AgencyLyfjastofnun Rebecca Sadun, MD, PhD Associate Professor, Medicine, Pediatrics, Dept of Medicine, Duke University School of Medicine Hermine I. Brunner, MD, MSc, MBA Professor of Pediatrics University of Cincinnati; Director, Division of Rheumatology at Cincinnati Children’s Hospital Richard Dimelow, PhD Director, Clinical Pharmacology at GSK, GlaxoSmithKline Jordan Roberts, MD, MPH Global Development Medical Associate Director, AstraZeneca Sule Yavuz, MD Global Clinical Program Lead, AstraZeneca Chloe Raymond Patient Representative	Presentation (PDF)
4:30 PM – 4:40 PM Closing Remarks	Speaker: Suzette Peng, MD Acting Clinical Team Leader, DRTM, CDER, FDA	Presentation (PDF)

Day 2 Workshop Details | July 31, 2026

Session 4: Trial Design Considerations (Efficacy & Safety)

9:00 AM – 9:10 AM

Opening Remarks

Speaker:

Lily Mulugeta, PharmD Associate Director, DPMH, CDER, FDA

Presentation (PDF)

9:10 AM – 9:25 AM

Safety Assessment in Pediatric SLE—Challenges and Opportunities

Speaker:

Mary Beth Son, MD Associate Professor, Clinical Chief, Division of
Immunology Section Chief, Rheumatology Program, Boston Children’s Hospital, Harvard Medical School

Presentation (PDF)

9:25 AM – 9:40 AM

Safety Assessment and Extrapolation in Pediatric SLE—Challenges and Opportunities

Speaker:

Laurie Conklin, MD Director, Child Health Innovation and Leadership
Department, Johnson & Johnson

Presentation (PDF)

9:40 AM – 10:15 AM

Alternative Approaches/Trial Design Considerations

Speakers:

Hermine I. Brunner, MD, MSc, MBA
Professor of Pediatrics University of Cincinnati; Director, Division of Rheumatology at Cincinnati Children’s
Hospital

Martine Dehlinger-Kremer, PhD, MS
Senior Development Strategy Lead Pediatrics, Special Patient Populations, UCB Biosciences

Presentation (PDF)

10:15 AM – 10:30 AM

Break

10:30 AM – 12:15 PM

Panel Discussion and Q/A (Future direction)

Moderator:
Raj Nair, MD
Division Director, DRTM, CDER, FDA

Panelists:

Lynne Yao, MD
Division Director, DPMH, CDER, FDA

Stacy Ardoin, MD
Professor, Pediatric and Adult Rheumatology,
Nationwide Children’s Hospital, The Ohio State University
College of Medicine

Hermine I. Brunner, MD, MSc, MBA
Professor of Pediatrics University of Cincinnati; Director, Division of Rheumatology at Cincinnati Children’s
Hospital

Stephen Balevic, MD, PhD, MHS Associate Professor, Departments of Medicine and Pediatrics, Duke University

Sarah Ringold, MD, MS Medical Director, Pediatric Development Team,
Immunology, Johnson & Johnson

Kalyanee Appanna, PhD Vice President, Global Head of Biostatistics, EMD Serono

Jordan Roberts, MD, MPH Global Development Medical Associate Director,
AstraZeneca

Caroline Benichou-Auriche, MD
EMA-Senior Clinical Expert, Icelandic Medicines AgencyLyfjastofnun

Nia Cooper
Patient Representative

Presentation (PDF)

12:15 PM – 12:30 PM

Closing Remarks

Speaker:

Raj Nair, MD
Division Director, DRTM, CDER, FDA

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